Successful Unrelated Umbilical Cord Blood Stem Cell Transplantation for Wiskott-Aldrich Syndrome in 2 Cases Report
Abstract: Purpose: To explore the clinical efficacy of unrelated umbilical cord blood stem cell transplantation for Wiskott-Aldrich syndrome. Method: Two patients were diagnosed with Wiskott-Aldrich syndrome by gene detection and received transplantation form unrelated umbilical cord blood stem cell. Two patients received a myelieblast conditioning with Cyclophosphamide, Busulfan, Fludarabine, Thiotepa. Cyclosporine A, Mycophenolate mofetil and Methotrexate were given to prophylaxis of graft versus host disease (GVHD). The doses of infused total nucleated cell (TNC) of two patients were 7.8 × 107/kg and 6.67 × 107/kg, respectively. The two patients’ CD34+ cells numbers were 8.32 × 104/kg and 6.67 × 104/kg, respectively. Result: The two patients had a successful engraftment, with 100% donor-derived cells by day 28 after transplantation. The time of absolute neutrophil count (ANC) > 0.5 × 109/L of two patients was 15 and 30 days, respectively. The time of platelet count > 20 × 109/L without transfusion was 30 and 45 days, respectively. Conclusion: The transplantation of umbilical cord blood stem cell from unrelated donor appears to be a feasible and effective strategy for further optimizing the outcome of HSCT for Wiskott-Aldrich syndrome.
文章引用: 陈丽白 , 温建芸 , 吴学东 (2016) 非亲缘脐血造血干细胞移植成功治疗WAS综合征2例报告。 亚洲儿科病例研究， 4， 5-9. doi: 10.12677/ACRP.2016.42002
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